日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

The manufacture of AAV for gene therapy applications using a closed, semi-automated hollow-fiber bioreactor.

利用封闭式半自动中空纤维生物反应器生产用于基因治疗的AAV

Soula Adrien, Leseigneur Florian, Anwar Amna, Ozdoganoglu Bilal, Gurung Jagan, Bhatti Hamza, Guenat Juline, Bazot Quentin, Sayed Majahar, Ricardo Carolina Pinto, Li Lily, Farukshina Katerina, Kheir Tony Bou, Mirmalek-Sani Hadi, Berger Gregory, Kerby Julie, Appleby Jonathan, Delahaye Michael

Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis

使用雷迪帕韦/索非布韦成功治疗一名患有由腺苷脱氨酶缺乏症引起的重症联合免疫缺陷病并合并丙型肝炎病毒感染的婴儿,使得使用Strimvelis进行基因治疗成为可能。

Tucci, Francesca; Calbi, Valeria; Barzaghi, Federica; Migliavacca, Maddalena; Ferrua, Francesca; Bernardo, Maria Ester; Canarutto, Daniele; Consiglieri, Giulia; Recupero, Salvatore; Calzatini, Francesco; Gabaldo, Michela; Lucano, Caterina; Casiraghi, Miriam; Darin, Silvia; Dionisio, Francesca; Marktel, Sarah; Cestone, Enza; Finazzi, Renato; Mieli-Vergani, Giorgina; Boeri, Enzo; Appleby, Jonathan; Abd Elaziz, Dalia; Ciceri, Fabio; Aiuti, Alessandro; Cicalese, Maria Pia

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

腺苷脱氨酶缺乏症基因治疗:短期和中期安全性的综合评估

Cicalese, Maria Pia; Ferrua, Francesca; Castagnaro, Laura; Rolfe, Katie; De Boever, Erika; Reinhardt, Rickey R; Appleby, Jonathan; Roncarolo, Maria Grazia; Aiuti, Alessandro

Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID

罕见病基因治疗:建立以患者为中心的Strimvelis治疗ADA-SCID患者登记库的益处与挑战

Stirnadel-Farrant, Heide; Kudari, Mahesh; Garman, Nadia; Imrie, Jessica; Chopra, Bikramjit; Giannelli, Stefania; Gabaldo, Michela; Corti, Ambra; Zancan, Stefano; Aiuti, Alessandro; Cicalese, Maria Pia; Batta, Rohit; Appleby, Jonathan; Davinelli, Mario; Ng, Pauline

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

关于逆转录病毒基因疗法治疗腺苷脱氨酶缺乏症所致免疫缺陷的安全性和有效性的最新进展

Cicalese, Maria Pia; Ferrua, Francesca; Castagnaro, Laura; Pajno, Roberta; Barzaghi, Federica; Giannelli, Stefania; Dionisio, Francesca; Brigida, Immacolata; Bonopane, Marco; Casiraghi, Miriam; Tabucchi, Antonella; Carlucci, Filippo; Grunebaum, Eyal; Adeli, Mehdi; Bredius, Robbert G; Puck, Jennifer M; Stepensky, Polina; Tezcan, Ilhan; Rolfe, Katie; De Boever, Erika; Reinhardt, Rickey R; Appleby, Jonathan; Ciceri, Fabio; Roncarolo, Maria Grazia; Aiuti, Alessandro